SB-007 (SpliceBio)

SB-007 is an investigational, first-in-class gene therapy developed by Splicebio for the treatment of Stargardt disease type 1 (STGD1). STGD1 is caused by mutations in the *ABCA4* gene, which encodes a large transmembrane protein essential for the visual cycle. Because the *ABCA4* cDNA (~6.8 kb) exceeds the standard packaging capacity of adeno-associated virus (AAV) vectors (~4.7 kb), SB-007 utilizes a proprietary Protein Splicing platform based on engineered split-inteins. The therapy consists of two separate AAV8 vectors: one encoding the N-terminal region and the other encoding the C-terminal region of the ABCA4 protein. Upon subretinal co-transduction of photoreceptor cells, the two protein fragments undergo trans-splicing to reconstitute the full-length, functional ABCA4 protein. This approach aims to restore retinoid transport, prevent the accumulation of toxic lipofuscin, and preserve vision in patients with STGD1.

Other names

AAV8 encoding ABCA4 C-regionAAV-8 encoding ABCA4 C-regionAAV 8 encoding ABCA4 C-regionAAV8 encoding ABCA4 N-regionAAV-8 encoding ABCA4 N-regionAAV 8 encoding ABCA4 N-regionABCA4 gene therapyABCA-4 gene therapyABCA 4 gene therapySplicebio ABCA4 therapy